PL1874306T3 - Kompozycje zawierające 1,2,4-oksadiazol aktywny po podaniu doustnie w celu supresyjnego leczenia niesensownej mutacji - Google Patents

Kompozycje zawierające 1,2,4-oksadiazol aktywny po podaniu doustnie w celu supresyjnego leczenia niesensownej mutacji

Info

Publication number
PL1874306T3
PL1874306T3 PL06749439T PL06749439T PL1874306T3 PL 1874306 T3 PL1874306 T3 PL 1874306T3 PL 06749439 T PL06749439 T PL 06749439T PL 06749439 T PL06749439 T PL 06749439T PL 1874306 T3 PL1874306 T3 PL 1874306T3
Authority
PL
Poland
Prior art keywords
oxadiazole
compositions
nonsense mutation
orally active
suppression therapy
Prior art date
Application number
PL06749439T
Other languages
English (en)
Polish (pl)
Inventor
Samit Hirawat
Langdon Miller
Original Assignee
Ptc Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ptc Therapeutics Inc filed Critical Ptc Therapeutics Inc
Publication of PL1874306T3 publication Critical patent/PL1874306T3/pl

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/4245Oxadiazoles
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/141Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/145Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic compounds
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    • A61P3/06Antihyperlipidemics
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
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    • A61P39/06Free radical scavengers or antioxidants
    • AHUMAN NECESSITIES
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    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • A61P5/10Drugs for disorders of the endocrine system of the posterior pituitary hormones, e.g. oxytocin, ADH
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    • AHUMAN NECESSITIES
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    • A61P9/00Drugs for disorders of the cardiovascular system
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    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis

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  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Diabetes (AREA)
  • Neurology (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurosurgery (AREA)
  • Endocrinology (AREA)
  • Biomedical Technology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Immunology (AREA)
  • Obesity (AREA)
  • Rheumatology (AREA)
  • Urology & Nephrology (AREA)
  • Pulmonology (AREA)
  • Oncology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Physiology (AREA)
  • Nutrition Science (AREA)
  • Transplantation (AREA)
  • Pain & Pain Management (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Child & Adolescent Psychology (AREA)
PL06749439T 2005-04-08 2006-04-06 Kompozycje zawierające 1,2,4-oksadiazol aktywny po podaniu doustnie w celu supresyjnego leczenia niesensownej mutacji PL1874306T3 (pl)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US66978905P 2005-04-08 2005-04-08
US78739506P 2006-03-30 2006-03-30
PCT/US2006/012887 WO2006110483A1 (en) 2005-04-08 2006-04-06 Compositions of an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy
EP06749439A EP1874306B1 (en) 2005-04-08 2006-04-06 Compositions of an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy

Publications (1)

Publication Number Publication Date
PL1874306T3 true PL1874306T3 (pl) 2012-11-30

Family

ID=36763092

Family Applications (2)

Application Number Title Priority Date Filing Date
PL11175569T PL2402002T3 (pl) 2005-04-08 2006-04-06 Kompozycje zawierające kwas 1,2,4-oksadiazolobenzoesowy i ich zastosowania do leczenia chorób związanych z przedwczesnym kodonem stop
PL06749439T PL1874306T3 (pl) 2005-04-08 2006-04-06 Kompozycje zawierające 1,2,4-oksadiazol aktywny po podaniu doustnie w celu supresyjnego leczenia niesensownej mutacji

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PL11175569T PL2402002T3 (pl) 2005-04-08 2006-04-06 Kompozycje zawierające kwas 1,2,4-oksadiazolobenzoesowy i ich zastosowania do leczenia chorób związanych z przedwczesnym kodonem stop

Country Status (16)

Country Link
US (7) US8716321B2 (pt)
EP (2) EP2402002B1 (pt)
JP (3) JP5800451B2 (pt)
CN (1) CN103720688A (pt)
AU (1) AU2006235115C1 (pt)
BR (1) BRPI0609089A2 (pt)
CA (1) CA2603402C (pt)
DK (2) DK2402002T3 (pt)
ES (2) ES2390804T3 (pt)
IL (2) IL186433A (pt)
MX (1) MX2007012206A (pt)
NO (2) NO339909B1 (pt)
NZ (1) NZ562197A (pt)
PL (2) PL2402002T3 (pt)
PT (2) PT2402002T (pt)
WO (1) WO2006110483A1 (pt)

Families Citing this family (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4101846B1 (en) 2003-04-11 2023-08-02 PTC Therapeutics, Inc. 1,2,4-oxadiazole benzoic acid compounds and their use for nonsense suppression and the treatment of disease
PL2402002T3 (pl) 2005-04-08 2018-11-30 Ptc Therapeutics, Inc. Kompozycje zawierające kwas 1,2,4-oksadiazolobenzoesowy i ich zastosowania do leczenia chorób związanych z przedwczesnym kodonem stop
EP2387995A1 (en) 2006-03-30 2011-11-23 PTC Therapeutics, Inc. Methods for the production of functional protein from DNA having a nonsense mutation and the treatment of disorders associated therewith
USRE50453E1 (en) 2006-04-07 2025-06-10 Vertex Pharmaceuticals Incorporated Indole derivatives as CFTR modulators
EP2674428B1 (en) 2006-04-07 2016-04-06 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
US10022352B2 (en) 2006-04-07 2018-07-17 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
US7645789B2 (en) 2006-04-07 2010-01-12 Vertex Pharmaceuticals Incorporated Indole derivatives as CFTR modulators
UA99265C2 (ru) 2006-09-08 2012-08-10 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способ получения 1,2,4-оксадиазолбензойной кислоты и ее производных (варианты)
US8101641B2 (en) 2006-09-25 2012-01-24 Ptc Therapeutics, Inc. Hydroxylated 1,2,4-oxadiazole benzoic acid compounds and compositions thereof
NZ575795A (en) * 2006-09-25 2012-03-30 Ptc Therapeutics Inc Crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid
MX2009003909A (es) * 2006-10-12 2009-05-25 Ptc Therapeutics Inc Métodos para dosificar un compuesto 1,2,4, - oxadiazol oralmente activo para el tratamiento de supresión de la mutación finalizadora.
AU2016262645B2 (en) * 2006-10-12 2018-05-17 Ptc Therapeutics, Inc. Methods for dosing an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy
US8563573B2 (en) 2007-11-02 2013-10-22 Vertex Pharmaceuticals Incorporated Azaindole derivatives as CFTR modulators
GB0715087D0 (en) 2007-08-03 2007-09-12 Summit Corp Plc Drug combinations for the treatment of duchenne muscular dystrophy
JP2010535708A (ja) 2007-08-03 2010-11-25 ビオマリン アイジーエー リミテッド デュシェンヌ型筋ジストロフィーの治療のための薬物併用
SI2578571T1 (sl) 2007-11-16 2016-01-29 Vertex Pharmaceuticals Incorporated Izokinolinski modulatorji prenašalcev z atp-vezavno kaseto
EP3683218B1 (en) 2007-12-07 2024-09-18 Vertex Pharmaceuticals Incorporated Solid forms of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl) benzoic acid
NZ616097A (en) 2008-03-31 2015-04-24 Vertex Pharma Pyridyl derivatives as cftr modulators
SI2826776T1 (sl) 2010-03-25 2021-02-26 Vertex Pharmaceuticals Incorporated Trdna disperzija amorfne oblike (R)-1(2,2-difluorobenzo(D)(1,3)dioksol-5-il)-N-(1-(2,3-dihidroksipropil) -6-fluoro-2-(1-hidroksi-2-metilpropan-2-il)-1H-indol-5-il)- ciklopropankarboksamida
US8802868B2 (en) 2010-03-25 2014-08-12 Vertex Pharmaceuticals Incorporated Solid forms of (R)-1(2,2-difluorobenzo[D][1,3]dioxo1-5-yl)-N-(1-(2,3-dihydroxypropyl-6-fluoro-2-(1-hydroxy-2-methylpropan2-yl)-1H-Indol-5-yl)-Cyclopropanecarboxamide
MX2012011655A (es) 2010-04-07 2012-11-23 Vertex Pharma Formas solidas de acido 3-(6-(1-(2,2-difluorobenzo [d][1-3]dioxol-5-il]ciclopropanocarboxamido)-3-metilpiridin-2-il) benzoico.
JP2013523833A (ja) 2010-04-07 2013-06-17 バーテックス ファーマシューティカルズ インコーポレイテッド 3−(6−(1−(2,2−ジフルオロベンゾ[d][1,3]ジオキソール−5−イル)シクロプロパンカルボキサミド)−3−メチルピリジン−2−イル)安息香酸の医薬組成物およびその投与
MX353408B (es) 2010-04-22 2018-01-11 Vertex Pharma Proceso para producir compuestos de cicloalquilcarboxamido-indol.
US8563593B2 (en) 2010-06-08 2013-10-22 Vertex Pharmaceuticals Incorporated Formulations of (R)-1-(2,2-difluorobenzo[D] [1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl)cyclopropanecarboxamide
ES2710854T3 (es) 2010-08-05 2019-04-29 Univ Lille Compuesto útil para el tratamiento de enfermedades mediadas por una mutación sin sentido y composición farmacéutica que comprende dicho compuesto
WO2012027247A2 (en) 2010-08-23 2012-03-01 Vertex Pharmaceuticals Incorporated Pharmaceutical composition of (r)-1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)-n-(1-(2,3-dihydroxy propyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1h-indol-5-yl) cyclopropanecarboxamide and administration therof
WO2013070961A1 (en) 2011-11-08 2013-05-16 Vertex Pharmaceuticals Incorporated Modulators of atp-binding cassette transporters
NZ727015A (en) 2012-01-25 2017-12-22 Vertex Pharma Formulations of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid
AR092857A1 (es) 2012-07-16 2015-05-06 Vertex Pharma Composiciones farmaceuticas de (r)-1-(2,2-difluorobenzo[d][1,3]dioxol-5-il)-n-(1-(2,3-dihidroxipropil)-6-fluoro-2-(1-hidroxi-2-metilpropan-2-il)-1h-indol-5-il)ciclopropancarboxamida y administracion de las mismas
EP2914248B2 (en) 2012-11-02 2023-10-18 Vertex Pharmaceuticals Incorporated Pharmaceutical compositions for the treatment of cftr mediated diseases
WO2014153643A1 (en) * 2013-03-26 2014-10-02 The University Of British Columbia Compositions and methods for use thereof in the treatment of aniridia
TWI695717B (zh) * 2014-03-06 2020-06-11 美商 Ptc 治療公司 1,2,4-二唑苯甲酸之鹽及醫藥組合物
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